EU orphan drugs regulation hailed a success by EBE and EuropaBio

The European Biopharmaceutical Enterprises (EBE) and EuropaBio’s Joint Rare Diseases and Orphan Medicinal Products Task Force released an assessment of the European Union’s Orphan Medicinal Products (OMP) Regulation evaluating the extent to which this legislation has impacted research into rare diseases and the availability of new medicines to treat rare diseases over the last 10 years.

The OMP regulation EC 141/2000, which came into force in April 2000, provided incentives for research, development and bringing to the market of medicines intended for the diagnosis, prevention or treatment of rare life-threatening or seriously debilitating conditions. Although individual rare diseases, which include certain cancers, metabolic diseases, diseases of the nervous system and musculoskeletal disorders, affect fewer than five in every 10,000 people, in combination, rare diseases may directly or indirectly affect an estimated 30 million people in Europe.

69 rare disease drugs now available, up from eight in 2000